Wednesday, June 07, 2023
Amphera B.V., a biotechnology company in the late stages of development, has made an announcement regarding its cell therapy called MesoPher. The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has recommended granting orphan medicinal product designation for MesoPher in the treatment of pancreatic cancer.
MesoPher is a cell therapy that involves autologous dendritic cells loaded with PheraLys, which is a lysate derived from tumor cell lines. This lysate contains various tumor-associated antigens, including those found in pancreatic cancer and other types of cancer.
In a phase II clinical trial called REACTIVE, conducted by Amphera in patients with resected pancreatic cancer, MesoPher demonstrated promising results. The trial showed a statistically significant 2-year Recurrence Free Survival rate of 60% and exhibited a favorable safety profile.
Orphan medicinal product designation is granted by the EMA to encourage the development of treatments for rare diseases. To be eligible for this designation, certain criteria must be met, such as addressing a life-threatening or chronically debilitating disease, having a low prevalence in the population, and providing significant additional benefits compared to existing treatments.
Receiving orphan designation provides companies with various incentives, including scientific advice, fee reductions, and access to EU grants, during the research and development phase. If a designated medicine successfully reaches the market and maintains the criteria, it is granted 10 years of market exclusivity.
It is important to note that orphan designation does not serve as an authorization, and not all designated medicines progress to the marketing authorization application stage. The evaluation process by the EMA's Committee for Medicinal Products for Human Use (CHMP) ensures that the medicine meets the rigorous standards for safety and efficacy required for all medicines.
